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Ionis/Akcea's ultra-rare disease drug rejected by FDA

Published on 28/08/18 at 11:45am

The FDA has opted to refuse approval to Akcea and Ionis’ Waylivra (volanesorsen) for the treatment of the ultra-rare hereditary condition familial chylomicronemia syndrome (FCS), despite the submission of Phase 3 data from the largest-ever study of the disease.

Both companies’ shares have plummeted on the news: Akcea’s share prices fell 21.5% while Ionis’ dropped by 14%.

The US regulator alerted the manufacturers via a complete response letter (CRL), originating from its Division of Metabolism and Endocrinology Products, but the reason for the rejection was not given.Submitted data had shown that Waylivra reduced triglycerides by 94% in patients compared to placebo, which raised levels by 18%

Regulators had raised earlier concerns about the low platelet count associated with the drug; it was expected that due to lack of available treatments the drug would be approved but there would be frequent monitoring of platelet levels.

FCS is characterised by extremely elevated triglyceride levels in the blood – levels which can’t be adequately metabolised due to a deficiency off lipoprotein lipase; it severely impacts daily life and can cause a range of damaging conditions including unpredictable and potentially fatal acute pancreatitis, chronic complications due to permanent organ damage.

“We are extremely disappointed with the FDA’s decision. FCS is an ultra-rare and debilitating disease. Our disappointment extends to the patient and physician community who currently do not have a treatment available to them,” commented Paula Soteropoulos, Chief Executive Officer of Akcea Therapeutics. “We continue to feel strongly that Waylivra demonstrates a favourable benefit/risk profile in people with FCS as was reflected in the positive outcome from our Advisory Committee hearing in May. We will continue to work with the FDA to confirm the path forward.”

Dr Brett P Monia, Chief Operating Officer of Ionis Pharmaceuticals, added: “We are fully supportive of WAYLIVRA and the many patients, physicians and researchers who are working to provide the first therapeutic option for FCS, a truly life-altering disease that deserves a treatment.”

US regulators are also currently evaluating a separate drug Tegsedi that has been developed by the companies. While Waylivra had estimated peak sales of $750 million by 2026, Tegsedi is expected to be an even bigger revenue earner. Tegsedi was approved in Europe in July with its label including platelet monitoring requirements due to the risk of low platelet count.

Matt Fellows

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